Ionis Expands Partnership with Sobi for Olezarsen Commercialisation Outside the U.S.
Thursday, March 27, 2025
Ionis Pharmaceuticals, Inc. has entered into a licence agreement granting Sobi exclusive rights to commercialise olezarsen outside the U.S., Canada, and China. Ionis will retain responsibility for olezarsen’s commercialisation in the U.S. The treatment was approved by the U.S. Food and Drug Administration (FDA) in December 2024 under the name TRYNGOLZA™ as an adjunct to diet for reducing triglycerides in adults with familial chylomicronemia syndrome (FCS). It is currently the only approved treatment for this rare genetic disease in the U.S.
Olezarsen is under review by the European Medicines Agency (EMA) for potential approval in treating FCS. Sobi will handle regulatory submissions and commercialisation in markets outside the U.S., except for Canada and China, where other licensing agreements are in place. Theratechnologies holds the licence for olezarsen’s commercialisation in Canada.
Sobi has extensive experience in commercialising treatments for rare and more common conditions, operating in over 30 countries. As Ionis’ current partner in Europe for Waylivra® (volanesorsen), the only approved FCS treatment in the region, Sobi aims to utilise its market knowledge and distribution network to support the introduction of olezarsen. The agreement includes an upfront payment to Ionis, milestone-based payments, and a tiered royalty reaching up to the mid-20% range on annual net sales.
Ionis is also evaluating olezarsen for severe hypertriglyceridemia (sHTG) through three Phase 3 clinical trials: CORE, CORE2, and ESSENCE. Data from ESSENCE are expected in mid-2025, while results from CORE and CORE2 are anticipated in the latter half of 2025.
FCS is a rare genetic condition that leads to extremely high triglyceride levels due to impaired function of the enzyme lipoprotein lipase (LPL). Individuals with FCS struggle to break down chylomicrons, which are lipoprotein particles composed mainly of triglycerides. The condition affects up to approximately 3,000 people in the U.S. and increases the risk of acute pancreatitis, chronic fatigue, and recurrent abdominal pain. It can also impact employment, adding to the disease burden.
sHTG is characterised by triglyceride levels of 500 mg/dL or higher and can result from genetic factors, diet, lifestyle, other medical conditions, or medications. Over three million people in the U.S. are estimated to have sHTG, which raises the risk of acute pancreatitis and atherosclerotic cardiovascular disease (ASCVD). Olezarsen has not yet been approved for sHTG treatment by any regulatory authority.
TRYNGOLZA™ (olezarsen) is an RNA-targeted therapy designed to lower the production of apoC-III, a liver-produced protein that plays a key role in triglyceride metabolism. It remains the only approved treatment for FCS in the U.S. and is intended to reduce triglycerides as part of dietary management.
Source: businesswire.com
